BREAKTHROUGHS BLOG

May 18, 2015

Research Roundup: Big Pharma’s role in antibiotic R&D, clinical trials for Ebola vaccines and drug-resistant tuberculosis treatments, and more

How can we get Big Pharma to invest in low-cost antibiotic research and development (R&D)? The Review of Antimicrobial Resistance—a research
group led by economist Jim O’Neill and established by United Kingdom Prime Minister David Cameron—proposed innovative answers to this question
in its recently released third report,
focused on the R&D pipeline for antibiotics. The report calls for the creation of a fund worth US$16 to 37 per decade to provide cash incentives of $2 to 3 billion for pharmaceutical companies that develop new antibiotics and agree to sell
them on a not-for-profit basis. Additionally, the report calls for the creation of a Global Antimicrobial Resistance Innovation Fund, worth $2 billion,
to support basic research into antibiotics and diagnostics that would enable the strategic use of antibiotics by primary care physicians. O’Neill reiterated
that the cost of inaction around antimicrobial resistance could be as high as $100 trillion and 10 million lives by 2050, as detailed in the first report.

A group of experts, including Executive Director Bernard Pécoul of the Drugs for Neglected Disease initiative—a member of the GHTC—are
calling on governments and other donors to establish
a pooled fund to address R&D gaps for antimicrobial resistance, emerging infectious diseases, and neglected diseases. The experts address the aforementioned
proposal to incentivize R&D for antibiotics, warning against a fragmented approach, and calling for coordination in not only financing, but throughout
the R&D process.

Liberia is now considered free from Ebola, after 42 days—twice the maximum incubation period—without a new case, and Guinea and Sierra Leone
reported only seven and two cases last week, respectively. As the West African epidemic comes to a close, clinical trials for experimental Ebola vaccines and drugs are underway.
The World Health Organization (WHO) hopes that it will be able to test the efficacy of the two drugs, Zmapp and sIRNA, but realizes that it may be
challenging to determine the efficacy of the two vaccine candidates, developed by GlaxoSmithKline and a partnership between Merck and NewLink Genetics.

Marie-Paule Kieny, assistant director-general for health systems and innovation at the WHO, noted that there are alternative forms of efficacy testing,
such as the “Animal Rule,” under which the
US Food and Drug Administration will approve a vaccine if it has proven safe for humans and effective in animals with similar immune systems as humans.
This rule can be applied for rare diseases or in cases where testing on humans would be unethical. The WHO also hosted an experts meeting on Ebola R&D last week, with the goal of creating a framework for expediting R&D in future outbreaks. While
Kieny highlighted recent progress, with drug and vaccine candidates for Ebola moving through the pipeline in months rather than years, an existing
framework would allow the WHO to respond rapidly.

TB Alliance, a member of GHTC, has initiated clinical trials of “Nix-TB,” the
first, all-oral drug regimen for extensively drug-resistant tuberculosis (XDR-TB). TB Alliance is sponsoring the trial in partnership with Janssen
Pharmaceutica—who provided a royalty-free license for one of the drugs in the regimen—and three hospitals in South Africa where Nix-TB
is being tested. The regimen is comprised of three drugs, including one that is currently undergoing clinical trials independent of Nix-TB and a general
antibiotic that has been used off-label to treat TB. There is currently no approved treatment for XDR-TB, and only 16 percent of patients receiving
treatment are cured after two years. According to TB Alliance President and CEO Dr. Mel Spigelman, “The launch of Nix-TB is a critical step to achieve
the vision of a truly short-course, simple, affordable, and well tolerated universal treatment regimen.”